DK2581448T3 - Tricyclo-DNA phosphorothioate - Google Patents
Tricyclo-DNA phosphorothioate Download PDFInfo
- Publication number
- DK2581448T3 DK2581448T3 DK11185129T DK11185129T DK2581448T3 DK 2581448 T3 DK2581448 T3 DK 2581448T3 DK 11185129 T DK11185129 T DK 11185129T DK 11185129 T DK11185129 T DK 11185129T DK 2581448 T3 DK2581448 T3 DK 2581448T3
- Authority
- DK
- Denmark
- Prior art keywords
- dna
- nucleic acid
- acid molecule
- mrna
- exon
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP11185129.1A EP2581448B1 (de) | 2011-10-13 | 2011-10-13 | Tricyclo-phosphorothioat-DNA |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2581448T3 true DK2581448T3 (en) | 2015-04-27 |
Family
ID=44799824
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK11185129T DK2581448T3 (en) | 2011-10-13 | 2011-10-13 | Tricyclo-DNA phosphorothioate |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US9738891B2 (de) |
| EP (2) | EP2581448B1 (de) |
| JP (1) | JP6181653B2 (de) |
| CN (1) | CN104245935B (de) |
| AU (1) | AU2012322903B2 (de) |
| BR (1) | BR112014009066B8 (de) |
| CA (2) | CA2776651C (de) |
| DK (1) | DK2581448T3 (de) |
| ES (2) | ES2535654T3 (de) |
| HK (1) | HK1186205A1 (de) |
| IL (1) | IL231983B (de) |
| IN (1) | IN2014DN03463A (de) |
| PL (1) | PL2581448T3 (de) |
| PT (1) | PT2581448E (de) |
| WO (1) | WO2013053928A1 (de) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7807816B2 (en) | 2004-06-28 | 2010-10-05 | University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| ES2852549T3 (es) | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| EP2350281B1 (de) | 2008-10-24 | 2014-05-14 | Sarepta Therapeutics, Inc. | Zusammensetzungen zum überspringen mehrerer exons bei dmd |
| HUE040445T2 (hu) | 2009-11-12 | 2019-03-28 | Univ Western Australia | Antiszensz molekulák és eljárások betegségek kezelésére |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| BR122020016865B1 (pt) | 2013-03-14 | 2022-12-27 | Sarepta Therapeutics, Inc. | Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd) |
| CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| EP3041935A1 (de) | 2013-09-05 | 2016-07-13 | Sage Therapeutics, Inc. | Antisense-induzierter exon2-einschluss in saurer alpha-glucosidase |
| EP3480312A1 (de) | 2013-09-11 | 2019-05-08 | Synthena AG | Nukleinsäuren und verfahren zur behandlung von morbus pompe |
| JP6872479B2 (ja) | 2014-07-31 | 2021-05-19 | アソシアシオン・アンスティテュ・ドゥ・ミオロジーＡｓｓｏｃｉａｔｉｏｎ Ｉｎｓｔｉｔｕｔ Ｄｅ Ｍｙｏｌｏｇｉｅ | 筋萎縮性側索硬化症の処置 |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| WO2016196670A1 (en) | 2015-06-01 | 2016-12-08 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type vii collagen |
| US20180237775A1 (en) | 2015-09-21 | 2018-08-23 | Association Institut De Myologie | Antisense oligonucleotides and uses thereof |
| BR112018007066A2 (pt) | 2015-10-09 | 2018-10-23 | Sarepta Therapeutics Inc | composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados |
| WO2017136435A1 (en) | 2016-02-01 | 2017-08-10 | The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health | Compounds for modulating fc-epsilon-ri-beta expression and uses thereof |
| JP7033547B2 (ja) | 2016-04-18 | 2022-03-10 | サレプタ セラピューティクス， インコーポレイテッド | 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法 |
| EA201892467A1 (ru) | 2016-04-29 | 2019-05-31 | Сарепта Терапьютикс, Инк. | Олигонуклеотидные аналоги, нацеленные на lmna человека |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| AU2017271524B2 (en) | 2016-05-24 | 2021-06-24 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| BR122022014984B1 (pt) | 2016-05-24 | 2024-03-05 | Sarepta Therapeutics, Inc | Compostos oligômeros morfolino fosforodiamidato |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45618A (fr) | 2016-06-30 | 2019-05-08 | Sarepta Therapeutics Inc | Sauts d'exons oligomères pour la dystrophie musculaire |
| WO2018055577A1 (en) | 2016-09-23 | 2018-03-29 | Synthena Ag | Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof |
| US11118179B2 (en) | 2016-09-23 | 2021-09-14 | Synthena Ag | Mixed tricyclo-DNA, 2′-modified RNA oligonucleotide compositions and uses thereof |
| HUE059843T2 (hu) | 2016-12-19 | 2023-01-28 | Sarepta Therapeutics Inc | Exonátugró oligomerkonjugátumok izomdisztrófiára |
| SG10202100491QA (en) | 2016-12-19 | 2021-02-25 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| CN110636866A (zh) | 2016-12-19 | 2019-12-31 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| CN110945005A (zh) * | 2017-04-20 | 2020-03-31 | 新特纳股份公司 | 包含三环dna核苷的修饰的寡聚化合物及其用途 |
| WO2018195338A1 (en) | 2017-04-20 | 2018-10-25 | Atyr Pharma, Inc. | Compositions and methods for treating lung inflammation |
| WO2018193428A1 (en) | 2017-04-20 | 2018-10-25 | Synthena Ag | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536058A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| WO2019067975A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| WO2019079637A2 (en) | 2017-10-18 | 2019-04-25 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERIC COMPOUNDS |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP4219717A3 (de) | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon-skipping-oligomere für muskeldystrophie |
| TW202020153A (zh) | 2018-07-27 | 2020-06-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| WO2020123574A1 (en) | 2018-12-13 | 2020-06-18 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP3955966A1 (de) | 2019-04-18 | 2022-02-23 | Sarepta Therapeutics, Inc. | Zusammensetzungen zur behandlung von muskeldystrophie |
| EP4054618A1 (de) | 2019-11-06 | 2022-09-14 | Association Institut de Myologie | Kombinationstherapie bei muskelerkrankungen |
| KR20220148230A (ko) | 2020-02-28 | 2022-11-04 | 아이오니스 파마수티컬즈, 인코포레이티드 | Smn2를 조절하기 위한 화합물 및 방법 |
| CA3171436A1 (en) | 2020-04-09 | 2021-10-14 | Association Institut De Myologie | Antisense sequences for treating amyotrophic lateral sclerosis |
| EP3978608A1 (de) | 2020-10-05 | 2022-04-06 | SQY Therapeutics | Oligomere verbindung zur dystrophinrettung in dmd-patienten während des skipping exon-51 |
| WO2023055774A1 (en) | 2021-09-30 | 2023-04-06 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
| WO2023070086A1 (en) | 2021-10-22 | 2023-04-27 | Sarepta Therapeutics, Inc. | Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases |
| WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| WO1999031262A2 (en) | 1997-12-16 | 1999-06-24 | Valentis, Inc. | Needle-free injection of formulated nucleic acid molecules |
| WO2000053722A2 (en) | 1999-03-10 | 2000-09-14 | Phogen Limited | Delivery of nucleic acids and proteins to cells |
| AU2004230927B9 (en) | 2003-04-13 | 2011-12-01 | Enzon Pharmaceuticals, Inc. | Polymeric oligonucleotide prodrugs |
| WO2007073149A1 (en) * | 2005-12-22 | 2007-06-28 | Keygene N.V. | Alternative nucleotides for improved targeted nucleotide exchange |
| MX2009002859A (es) * | 2006-09-15 | 2009-03-30 | Enzon Pharmaceuticals Inc | Enlazadores biodegradables a base de ester impedido para suministro de oligonucleotidos. |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| ES2694726T3 (es) * | 2007-06-29 | 2018-12-26 | Sarepta Therapeutics, Inc. | Conjugados peptídicos específicos de tejido y métodos |
| JP2012509366A (ja) * | 2008-11-17 | 2012-04-19 | エンゾン ファーマシューティカルズ，インコーポレーテッド | 核酸送達系のための放出可能ポリマー脂質 |
| WO2010115993A1 (en) * | 2009-04-10 | 2010-10-14 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
| CN103477273A (zh) | 2011-02-25 | 2013-12-25 | 卢米泰克斯公司 | 显示器前方照明装置 |
-
2011
- 2011-10-13 PL PL11185129T patent/PL2581448T3/pl unknown
- 2011-10-13 PT PT111851291T patent/PT2581448E/pt unknown
- 2011-10-13 DK DK11185129T patent/DK2581448T3/en active
- 2011-10-13 ES ES11185129.1T patent/ES2535654T3/es active Active
- 2011-10-13 EP EP11185129.1A patent/EP2581448B1/de active Active
-
2012
- 2012-04-27 CA CA2776651A patent/CA2776651C/en active Active
- 2012-10-12 ES ES12770503.6T patent/ES2651216T3/es active Active
- 2012-10-12 CA CA2851970A patent/CA2851970A1/en not_active Abandoned
- 2012-10-12 WO PCT/EP2012/070349 patent/WO2013053928A1/en active Application Filing
- 2012-10-12 US US14/351,733 patent/US9738891B2/en active Active
- 2012-10-12 AU AU2012322903A patent/AU2012322903B2/en active Active
- 2012-10-12 BR BR112014009066A patent/BR112014009066B8/pt active IP Right Grant
- 2012-10-12 JP JP2014535113A patent/JP6181653B2/ja active Active
- 2012-10-12 CN CN201280057688.6A patent/CN104245935B/zh active Active
- 2012-10-12 EP EP12770503.6A patent/EP2766479B1/de active Active
-
2013
- 2013-10-15 HK HK13111544.3A patent/HK1186205A1/xx unknown
-
2014
- 2014-04-07 IL IL231983A patent/IL231983B/en active IP Right Grant
- 2014-04-30 IN IN3463DEN2014 patent/IN2014DN03463A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP2766479B1 (de) | 2017-09-13 |
| IN2014DN03463A (de) | 2015-06-05 |
| EP2581448A1 (de) | 2013-04-17 |
| PL2581448T3 (pl) | 2015-08-31 |
| CA2776651C (en) | 2021-06-08 |
| BR112014009066B8 (pt) | 2021-02-23 |
| ES2535654T3 (es) | 2015-05-13 |
| US9738891B2 (en) | 2017-08-22 |
| BR112014009066B1 (pt) | 2020-08-25 |
| JP2015501144A (ja) | 2015-01-15 |
| JP6181653B2 (ja) | 2017-08-16 |
| EP2581448B1 (de) | 2015-01-28 |
| CA2776651A1 (en) | 2013-04-13 |
| PT2581448E (pt) | 2015-05-21 |
| CN104245935A (zh) | 2014-12-24 |
| ES2651216T3 (es) | 2018-01-25 |
| BR112014009066A8 (pt) | 2018-01-09 |
| CN104245935B (zh) | 2018-05-08 |
| WO2013053928A1 (en) | 2013-04-18 |
| EP2766479A1 (de) | 2014-08-20 |
| AU2012322903B2 (en) | 2017-09-14 |
| IL231983B (en) | 2018-04-30 |
| HK1186205A1 (en) | 2014-03-07 |
| AU2012322903A1 (en) | 2014-05-29 |
| IL231983A0 (en) | 2014-05-28 |
| BR112014009066A2 (pt) | 2017-04-18 |
| CA2851970A1 (en) | 2013-04-18 |
| US20140296323A1 (en) | 2014-10-02 |
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